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| |  Researchers Using Gene Therapy To Protect Human Cells From HIV FARMINGDALE, N.Y., -- April 17, 1997 -- Scientists have successfully used gene therapy in the laboratory to produce human immune (CD4+) cells with resistance to repeated challenges by HIV over a prolonged period, it was reported in the May edition of the Journal of Virology, the peer-reviewed journal of the American Society for Microbiology. The more than year-long study, showed that CD4+ monocyte cells, which are normally sensitive to HIV, are protected from HIV infection when treated with this new form of gene therapy, known as genetic antisense. The study was conducted by Enzo Biochem, a New York-based biotechnology company and researchers including Jeffrey Laurence, MD from New York Hospital-Cornell University Medical College. "This is a very important step in moving towards the development of an effective clinical product with wide applications for HIV therapy. The use of genetic antisense for HIV, either alone or in combination with already established protocols, can become a powerful weapon in the fight against AIDS," said Dr. Dean Engelhardt, Senior Vice President of Research, Enzo Biochem. Enzo Biochem is in the process of petitioning the FDA to begin human clinical trials of their genetic antisense technology on HIV infected patients. Genetic antisense is a technology that uses specially constructed genes inserted directly into cells to regulate the function of target genes. Thus, the genes that HIV requires to grow and propagate in immune cells can be "turned off" with this technology. In the Journal of Virology paper, the researchers describe how they were able to insert a special construct containing three different antisense genes into immune cells to make them resistant to repeated exposures to HIV. The multi-targeting approach was designed to combat the high rate of variability and mutability of HIV by creating three independent target sequences. A key element in the success of the study was the development by Enzo scientists of the construct used in these studies, which was designed to localize primarily in the cell nucleus where it can be most effective in inhibiting viral growth. The construct was also designed to be "invisible" to the human immune system, so as not to trigger an immune response. "While current anti-HIV therapy, including the use of multiple combinations of drugs such as AZT and protease inhibitors, has demonstrated clinical benefit in many patients, this therapy has limitations. Resistance to the drugs has developed in certain populations of patients and failure to take medications as prescribed may increase the likelihood of drug resistance," said Dr. Engelhardt. "Since the antisense genes are designed to be incorporated into a patient's susceptible cells and to function continuously, compliance to a therapeutic regimen by the patient may be eliminated." Enzo Biochem has been engaged in the development and research of genetic antisense and other gene regulation proprietary technologies for more than a decade.
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